Clinical trials: Genetic Mutation
Prostatic Neoplasms
Prevalence of HRR-related Genes Mutations and Prognosis in Metastatic Castration Resistant Prostate Cancer (mCRPC) Patients in Real World Setting
The purpose of this study is to investigate the prevalence of tissue homologous recombination repair (HRR)-related gene mutations (positive/negative/Variant of uncertain significance (VUS)), clinical outcome such as…
- Trial name
- ZENSHIN (NCT04425200)
- Where
- 26 sites in Japan
- Volunteers
- 205 people
- Sponsor
- AstraZeneca
Primary Open Angle Glaucoma
CRISPR/Cas9 Instantaneous Gene Editing Therapy to Intraocular Hypertensive POAG With MYOC Mutation
This study is intented to evaluate the safety, tolerability and preliminary efficacy of CRISPR/Cas9 Instantaneous Gene Editing Therapy (BD113 virus-like particle, also BD113vLVP) in patients with primary open-angle glaucoma…
- Trial name
- NCT06465537
- Testing
- BD113vVLP
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Solid Tumor
A PhaseI Study of HL-085 Plus Vemurafenib in Solid Tumor With BRAF V600 Mutation
This is a phase I, open-label, dose escalation study to evaluate tolerability, safety, pharmacokinetics and efficacy in patients with BRAF V600 mutant advanced solid tumor by HL-085 plus Vemurafenib treatment.
- Trial name
- NCT03781219
- Testing
- HL-085, Vemurafenib
- Where
- 4 sites in China
- Volunteers
- 45 people
- Sponsor
- Shanghai Kechow Pharma, Inc.
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 52 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
Duchenne Muscular Dystrophy
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD.
- Trial name
- NCT03648827
- Testing
- Ataluren
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- PTC Therapeutics
Cancer
A Study to Evaluate the Effect of Repeat Oral Dosing of GSK2118436 on Cardiac Repolarization in Subjects With V600 BRAF Mutation-Positive Tumors
This is a Phase I, multicenter, 2-part study with Part 1 designed as a safety lead-in and Part 2 designed to evaluate the effect of GSK2118436 on cardiac repolarization (corrected QT interval [QTc] duration) as compared with…
- Trial name
- NCT01738451
- Testing
- GSK2118436 75 mg
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 50 people
- Sponsor
- GlaxoSmithKline
Carcinoma, Non-Small-Cell Lung
A Study of Adagrasib Plus Pembrolizumab Plus Chemotherapy vs. Placebo Plus Pembrolizumab Plus Chemotherapy in Participants With Previously Untreated Non-squamous Non-small Cell Lung Cancer With KRAS G12C Mutation (KRYSTAL-4)
This is a trial to evaluate the efficacy, safety, and tolerability of adagrasib plus pembrolizumab plus platinum-doublet chemotherapy versus placebo plus pembrolizumab plus platinum-doublet chemotherapy in participants with…
- Trial name
- NCT06875310
- Testing
- Adagrasib, Pembrolizumab, Carboplatin
- Where
- 317 sites in United States, Argentina and 33 more
- Volunteers
- 630 people
- Sponsor
- Mirati Therapeutics Inc.
RAS Mutation, Advanced Solid Tumor
A First-in-Human Study Investigating BG-85738 Alone or in Combination With Other Antitumor Agents in Patients With Advanced or Metastatic Solid Tumors With Rat Sarcoma Virus (RAS) Mutations
The purpose of this study is to test if the BG-85738 is safe and if it works in patients with advanced solid tumors with RAS mutations when it is given on its own and in combination.
- Conditions
- RAS Mutation, Advanced Solid Tumor, Metastatic Solid Tumor
- Trial name
- NCT07746141
- Testing
- BG-85738, Tislelizumab, Cetuximab
- Where
- 3 sites in Australia, New Zealand
- Volunteers
- 100 people
- Sponsor
- BeOne Medicines
Locally Advanced or Metastatic Non-Small Cell Lung Cancer, EGFR Exon20 Insertion Mutations
Real-World Effectiveness of Osimertinib for Patients With Non-Small Cell Lung Cancer
This study is a retrospective real-world study to evaluate the efficacy of Osimertinib for patients with advanced or metastatic EGFR 20exon insertion mutation (20ins) Non-Small Cell Lung Cancer.
- Trial name
- NCT05513664
- Testing
- Osimertinib
- Where
- 24 sites in China
- Volunteers
- 80 people
- Sponsor
- Shanghai JMT-Bio Inc.
Non-Small Cell Lung Cancer, NSCLC, KRAS, NRAS, HRAS-mutated NSCLC
Study of RAS(ON) Inhibitors in Patients With Advanced RAS-mutated NSCLC
The purpose of this platform study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary antitumor activity of novel RAS(ON) inhibitors as a monotherapy or combined with Standard(s) of Care (SOC) or with…
- Conditions
- Non-Small Cell Lung Cancer, NSCLC, KRAS, NRAS, HRAS-mutated NSCLC, KRAS G12C-mutated Solid Tumors, Lung Cancer, Lung Cancer Stage IV, Advanced Solid Tumor, Cancer
- Trial name
- NCT06162221
- Testing
- RMC-6291, RMC-6236, Pembrolizumab
- Where
- 98 sites in United States, Australia and 11 more
- Volunteers
- 616 people
- Sponsor
- Revolution Medicines, Inc.
Cystic Fibrosis
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)
This is a Phase 3, 2-part (Part A and Part B), open label, multicenter study evaluating the pharmacokinetic (PK), safety, and tolerability of multiple doses of tezacaftor (TEZ) in combination with ivacaftor (IVA) in subjects 6…
- Trial name
- NCT02953314
- Testing
- TEZ, TEZ/IVA, IVA
- Where
- 33 sites in United States, Canada
- Volunteers
- 83 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Advanced Solid Tumors, Breast Cancer Recurrent
First-in-Human Study of ATX-559, an Oral Inhibitor of DHX9, in Patients With Advanced or Metastatic Solid Tumors, and Molecularly Defined Cancers
The goal of this study is to identify a safe and tolerated dose of the orally administered DHX9 inhibitor ATX-559. In addition, this study will evaluate the pharmacokinetics, pharmacodynamics and preliminary antitumor activity of…
- Conditions
- Advanced Solid Tumors, Breast Cancer Recurrent, Colorectal Cancer Metastatic, Colon Cancer
- Trial name
- NCT06625515
- Testing
- ATX-559
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Accent Therapeutics
Non-Small Cell Lung Cancer
Study For Patients With NSCLC EGFR Mutations (Del 19 or L858R +/- T790M)
This is a Phase 1/2 study of PF-06747775 as a single agent and in combination with other cancer treatments in patients with advanced EGFRm NSCLC.
- Trial name
- NCT02349633
- Testing
- PF-06747775, Palbociclib, Avelumab
- Where
- 15 sites in United States, Australia and 2 more
- Volunteers
- 65 people
- Sponsor
- Pfizer
Dystrophic Epidermolysis Bullosa
Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene
A double-blind, randomized, intra-patient placebo- controlled, multiple dose study of PTW-002 evaluating safety, proof of mechanism, preliminary efficacy, and systemic exposure in patients with Dominant Dystrophic Epidermolysis…
- Trial name
- NCT05529134
- Testing
- PTW-002 10 mg/g gel
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Phoenicis Therapeutics
Advanced Solid Tumor, Unresectable Solid Tumor
A Beta-only IL-2 ImmunoTherapY Study
This is a Phase 1/2, multi-center, open-label, dose-escalation and expansion study to evaluate safety and tolerability, PK, pharmacodynamic, and early signal of anti-tumor activity of MDNA11 alone or in combination with a…
- Conditions
- Advanced Solid Tumor, Unresectable Solid Tumor, Clear Cell Renal Cell Carcinoma, Triple Negative Breast Cancer
- Trial name
- ABILITY-1 (NCT05086692)
- Testing
- MDNA11, Pembrolizumab (KEYTRUDA®)
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 160 people
- Sponsor
- Medicenna Therapeutics, Inc.
Non-Small Cell Lung Cancer
A Study of SYS6010 Combined With Osimertinib Versus Osimertinib Alone as Neoadjuvant Therapy for Patients With EGFR Mutation-positive Resectable Non-squamous Non-small Cell Lung Cancer
To evaluate the safety and efficacy of SYS6010 combined with osimertinib as neoadjuvant therapy for patients with resectable EGFR mutation non-squamous non-small cell lung cancer.
- Trial name
- NCT07256509
- Testing
- SYS6010, Osimertinib, Pemetrexed
- Volunteers
- 120 people
- Sponsor
- CSPC Megalith Biopharmaceutical Co.,Ltd.
Cystic Fibrosis
PTC124 for Cystic Fibrosis
In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein.
- Trial name
- NCT00234663
- Testing
- PTC124
- Where
- 5 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Glioblastoma
Adult Study: ABT-414 Alone or ABT-414 Plus Temozolomide vs. Lomustine or Temozolomide for Recurrent Glioblastoma Pediatric Study: Evaluation of ABT-414 in Children With High Grade Gliomas
This study was conducted to evaluate the efficacy and safety of depatuxizumab mafodotin (ABT-414) alone or with temozolomide versus temozolomide or lomustine alone in adult participants with recurrent glioblastoma.
- Trial name
- INTELLANCE-2 (NCT02343406)
- Testing
- Depatuxizumab mafodotin, Temozolomide, Lomustine
- Where
- 102 sites in United States, Australia and 19 more
- Volunteers
- 266 people
- Sponsor
- AbbVie
Crohn's Disease, Ulcerative Colitis (UC)
A Blood and Tissue Sample Collection Study of Patients Who Have Inflammatory Bowel Disease, Who Have Been Treated With Adalimumab and Who Developed Hepatosplenic T-Cell Lymphoma
To collect and store blood and biopsy samples obtained from CD or UC patients exposed to adalimumab and diagnosed with Hepatosplenic T-cell Lymphoma (HSTCL), for the purpose of identifying potential biomarkers and genetic…
- Conditions
- Crohn's Disease, Ulcerative Colitis (UC), Hepatosplenic T-Cell Lymphoma
- Trial name
- HSTCL (NCT02087878)
- Where
- Italy
- Sponsor
- AbbVie
Breast Cancer
Study of Safety and Efficacy of Dapagliflozin + Metformin XR Versus Metformin XR in Participants With HR+, HER2-, Advanced Breast Cancer While on Treatment With Alpelisib and Fulvestrant
This study was designed to assess the safety and efficacy of the combination of dapagliflozin plus metformin extended release (XR) compared with metformin XR during treatment with alpelisib plus fulvestrant in participants with…
- Trial name
- EPIK-B4 (NCT04899349)
- Testing
- Alpelisib, Fulvestrant, Metformin XR
- Where
- 2 sites in United States, Malaysia
- Volunteers
- 2 people
- Sponsor
- Novartis Pharmaceuticals
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